Time course of Amyotrophic Lateral Sclerosis Functional Rating Scale, Quality of life as measured by the EQ-5D rating scale [ Time Frame: Multiple assessments over 3 months ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ICMJE

Same as current

Current Other Outcome Measures ICMJE

Not Provided

Original Other Outcome Measures ICMJE

Not Provided

Descriptive Information

Brief Title ICMJE

A Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis

Official Title ICMJE

A Double-blind, Randomised, Parallel Group Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis, Using an Implanted Catheter and SynchroMed® II Pump

Brief Summary

This study is conducted to evaluate the safety and tolerability of the drug product sNN0029, containing the growth factor VEGF165, when administered directly into one of the fluid filled cavities in the brain using an implanted catheter and an implanted SynchroMed® II pump. Patients with Amyotrophic Lateral Sclerosis will be enrolled.

Detailed Description

Vascular endothelial growth factor (VEGF) is an endogenous human protein fundamental to the development of the vascular and nervous systems in the body. A role for VEGF in ALS has been suggested from observations in animal models of the disease as well as observations of a dysregulation of VEGF production in patients with ALS.

NeuroNova intends to investigate whether intracerebroventricular administration of VEGF165 in the form of the drug product sNN029 can improve motor function and prolong survival in patients with ALS, and in this first study the safety and tolerability of treatment for 3 months will be evaluated.

Possible signs of intracranial bleeding or loss of blood-brain-barrier integrity through measurements of bilirubin and albumin levels in cerebrospinal fluid collected through lumbar and cervical puncture

MRI/magnetic resonance angiography (MRA) examination of the brain and cervical spinal cord at screening with no findings of tumors or potential sources of pathological bleedings, or abnormality that may interfere with the assessments of safety or efficacy or that would, in the judgement of the investigator, represent a surgical risk to the subject.

Patient is medically able to undergo the surgery required for stereotactic implantation of the catheter and infusion pump.

Patient has been given written and verbal information, has had the opportunity to ask questions about the study, and understands the time and procedural commitments.

Patient has given signed consent (written) to participate in the study. In the event that a patient who gives oral informed consent is not physically able to sign the informed consent form (ICF) due to disease progression, a witness may sign the ICF on the patient's behalf.

Exclusion Criteria:

Impaired respiratory function judged to pose a risk to the patient during anaesthesia for the device implantation.

Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally. Physicians should specifically investigate anatomical factors at or near the implant site (e.g., vascular abnormalities, neoplasms, or other abnormalities), underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., haemophilia, Von Willebrand's disease, liver disease, or other medical conditions), and the administration of any antiplatelet or anticoagulant medication (e.g., aspirin, Plavix, NSAIDs) in the pre- or perioperative period. Any of those conditions or drugs could place a patient at an increased risk for intraoperative or postoperative bleeding.

Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter.

Presence of cardiac pacemakers, spinal cord stimulators, implantable programmable intraspinal drug pumps, or any other device that may interfere or interact with the programmer, without prior approval by Medtronic.

Clinically significant abnormalities in hematology or clinical chemistry parameters as assessed by the investigator.

Ongoing medical condition that according to the investigator would interfere with the conduct and assessments in the study. Examples are medical disability (e.g., severe degenerative arthritis, compromised nutritional state, peripheral neuropathy) that would interfere with the assessment of safety and efficacy of investigational product or device performance, or would compromise the ability of the subject to undergo study procedures (e.g., MRI), or to give informed consent.

Participation in another clinical trial with an investigational drug or device within 3 months prior to Screening visit.

For female subjects, ongoing pregnancy or planned pregnancy during the period of treatment with study drug.

Breast feeding during the period of treatment with study drug.

Gender

Both

Ages

18 Years to 75 Years (Adult, Senior)

Accepts Healthy Volunteers

No

Contacts ICMJE

Contact information is only displayed when the study is recruiting subjects